RECRUITING

Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms

Conditions

Myelodysplastic Syndromes Myeloproliferative Neoplasm Anemia MDS MPN Non-Proliferative MDS Non-Proliferative MPN

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of the study is to see if participants with anemia due to their type of MDS or MDS/MPN will experience a more decreased need for regular blood transfusions if they take luspatercept plus best supportive care, and what effect, good and/or bad, luspatercept has on them and their anemia due to MDS or MDS/MPN. The safety and tolerability of luspatercept will also be evaluated in this study.

Official Title

A Phase 2, Single Arm Study of Luspatercept for the Treatment of Anemia in Lower Risk Myelodysplastic Syndromes (MDS) or Non-Proliferative Myelodysplastic Syndromes/ Myeloproliferative Neoplasms (MDS/MPN)

Quick Facts

Study Start:2023-02-21

Study Completion:2026-09

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05732961

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Participant is ≥18 years at the time of signing the informed consent form 2. Participant is willing and able to adhere to the study visit schedule and other protocol requirements 3. Documented diagnosis of MDS or non-proliferative MDS/MPN (WBC \< 13,000 U/L) 1. According to WHO 2016 classification 2. Meets IPSS-R classification of very low, low, or intermediate risk disease 4. Documented acquired splicing gene mutation 1. Cohort 1: detectable splicing mutation other than SF3B1: (SRSF2, U2AF1, ZRSR2) 2. Cohort 2: SF3B1 mutation with prior treatment with hypomethylating agent and or lenalidomide 5. \<5% blasts in bone marrow 6. Refractory, intolerant to, or ineligible for, prior ESA treatment, as defined by any one of the following: 1. Refractory to prior ESA treatment - non-response or response that is no longer maintained. ESA regimen must have been either: * rHu EPO ≥ 40,000 IU/wk for at least 8 doses or equivalent Or darbepoetin alpha ≥ 500 μg Q3W for at least 4 doses or equivalent 2. Intolerant to prior ESA treatment - discontinuation of prior ESA-containing regimen, at any time after introduction due to intolerance or AE 3. ESA ineligible - Low chance of response to ESA based on endogenous serum EPO \> 200 U/L for subjects not previously treated with ESAs 7. Discontinuation of ESAs, G-CSF, GM-CSF ≥ 4 weeks prior to start of study treatment 8. Require RBC transfusions 9. Applies to on treatment subjects only - females of childbearing potential (FCBP) defined as a sexually mature woman who: 1. has achieved menarche at some point, 2. has not undergone a hysterectomy or bilateral oophorectomy, or 3. has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) and must: * Have two negative pregnancy tests 48 hours apart as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy. This applies even if the subject practices true abstinence\* from heterosexual contact. * Either commit to true abstinence\from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting 10. investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy 11. Applies to on treatment subjects only - Male subjects must: 1. Practice true abstinence\ (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 84 days following investigational product discontinuation even if he has undergone a successful vasectomy. \* True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence \[eg, calendar, ovulation, symptothermal, post-ovulation methods\] and withdrawal are not acceptable methods of contraception).	1. Prior allogeneic or autologous stem cell transplant 2. MDS associated with del 5q cytogenetic abnormality if no prior lenalidomide treatment 3. Uncontrolled hypertension, defined as repeated elevations of diastolic blood pressure (DBP) ≥ 100 mmHg despite adequate treatment 4. ANC \< 500/μL (0.5 x 109/L) 5. Platelet count ˂50,000/μL (50 x 109/L) 6. Active other malignancies 7. Severe renal impairment (eGFR \< 30 mL/min/1.73 m2) 8. ALT or AST ≥ 3 × ULN 9. Prior treatment with Luspatercept or Sotatercept 10. Pregnant or breastfeeding females

Inclusion Criteria

Exclusion Criteria

1. Participant is ≥18 years at the time of signing the informed consent form
2. Participant is willing and able to adhere to the study visit schedule and other protocol requirements
3. Documented diagnosis of MDS or non-proliferative MDS/MPN (WBC \< 13,000 U/L)
1. According to WHO 2016 classification
2. Meets IPSS-R classification of very low, low, or intermediate risk disease
4. Documented acquired splicing gene mutation
1. Cohort 1: detectable splicing mutation other than SF3B1: (SRSF2, U2AF1, ZRSR2)
2. Cohort 2: SF3B1 mutation with prior treatment with hypomethylating agent and or lenalidomide
5. \<5% blasts in bone marrow
6. Refractory, intolerant to, or ineligible for, prior ESA treatment, as defined by any one of the following:
1. Refractory to prior ESA treatment - non-response or response that is no longer maintained. ESA regimen must have been either:
* rHu EPO ≥ 40,000 IU/wk for at least 8 doses or equivalent Or darbepoetin alpha ≥ 500 μg Q3W for at least 4 doses or equivalent
2. Intolerant to prior ESA treatment - discontinuation of prior ESA-containing regimen, at any time after introduction due to intolerance or AE
3. ESA ineligible - Low chance of response to ESA based on endogenous serum EPO \> 200 U/L for subjects not previously treated with ESAs
7. Discontinuation of ESAs, G-CSF, GM-CSF ≥ 4 weeks prior to start of study treatment
8. Require RBC transfusions
9. Applies to on treatment subjects only - females of childbearing potential (FCBP) defined as a sexually mature woman who:
1. has achieved menarche at some point,
2. has not undergone a hysterectomy or bilateral oophorectomy, or
3. has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) and must:
* Have two negative pregnancy tests 48 hours apart as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy. This applies even if the subject practices true abstinence\* from heterosexual contact.
* Either commit to true abstinence\*from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting
10. investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy
11. Applies to on treatment subjects only - Male subjects must:
1. Practice true abstinence\* (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 84 days following investigational product discontinuation even if he has undergone a successful vasectomy. \* True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence \[eg, calendar, ovulation, symptothermal, post-ovulation methods\] and withdrawal are not acceptable methods of contraception).

1. Prior allogeneic or autologous stem cell transplant
2. MDS associated with del 5q cytogenetic abnormality if no prior lenalidomide treatment
3. Uncontrolled hypertension, defined as repeated elevations of diastolic blood pressure (DBP) ≥ 100 mmHg despite adequate treatment
4. ANC \< 500/μL (0.5 x 109/L)
5. Platelet count ˂50,000/μL (50 x 109/L)
6. Active other malignancies
7. Severe renal impairment (eGFR \< 30 mL/min/1.73 m2)
8. ALT or AST ≥ 3 × ULN
9. Prior treatment with Luspatercept or Sotatercept
10. Pregnant or breastfeeding females

Contacts and Locations

Study Contact

Rami Komrokji, MD

CONTACT

813-745-4748

Rami.Komrokji@moffitt.org

Principal Investigator

Rami Komrokji, MD

PRINCIPAL_INVESTIGATOR

Moffitt Cancer Center

Study Locations (Sites)

Moffitt Cancer Center

Tampa, Florida, 33612

United States

Collaborators and Investigators

Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Rami Komrokji, MD, PRINCIPAL_INVESTIGATOR, Moffitt Cancer Center

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-02-21

Study Completion Date2026-09

Study Record Updates

Study Start Date2023-02-21

Study Completion Date2026-09

Terms related to this study

Keywords Provided by Researchers

MDS
MPN
Non-Proliferative MDS
Non-Proliferative MPN

Additional Relevant MeSH Terms

Myelodysplastic Syndromes
Myeloproliferative Neoplasm
Anemia